Homology Medicines Presents Data on Platform’s Ability to Target the Central and Peripheral Nervous System and Therapeutic Potential in the Rare Disease Metachromatic Leukodystrophy
Homology to Nominate a Clinical Development Candidate for MLD This Quarter and to Begin IND-Enabling Studies in 2019
“The key requirement for developing single IV gene therapies for neurological disorders is the ability for the therapeutic to cross the blood-brain-barrier, and we are pleased to report that our vectors effectively reach the central and peripheral nervous system following systemic administration,” said
For more information about this presentation, visit Homology’s website at www.homologymedicines.com/publications.
About Metachromatic Leukodystrophy (MLD)
MLD is a rare lysosomal storage disorder caused by a mutation in the ASA gene. ASA is responsible for the creation of the arylsulfatase A (ARSA) protein, which is required for the breakdown of cellular components. In MLD, these cellular components accumulate and destroy myelin-producing cells in the peripheral and central nervous system leading to progressive and serious neurological deterioration. The late infantile form of the disorder is estimated to affect 1 in 40,000 people, and it is fatal within 5-10 years after onset.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including statements regarding anticipated timing for nominating an MLD development candidate and initiating IND-enabling studies; advancing our novel gene therapy and gene editing technology platform and pipeline; our beliefs regarding our manufacturing capabilities; our goal of improving the lives of patients with rare genetic diseases; the anticipated timing of the release of clinical data; beliefs about preclinical data; and our position as a leader in the development of genetic medicines. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the fact that we have and expect to continue to incur significant losses; our need for additional funding, which may not be available; failure to identify additional product candidates and develop marketable products; the early stage of our development efforts; our failure or the failure of our collaborators to successfully develop and commercialize drug candidates; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the build out of our manufacturing facility; risks relating to the regulatory approval process; our product candidates may cause serious adverse side effects; inability to maintain our collaborations, or the failure of these collaborations; our reliance on third parties; the inability to obtain orphan drug exclusivity; failure to obtain international marketing approval; failure to obtain U.S. marketing approval; ongoing regulatory obligations; effects of significant competition; unfavorable pricing regulations, third-party reimbursement practices or healthcare reform initiatives; product liability lawsuits; failure to attract, retain and motivate qualified personnel; the possibility of system failures or security breaches; risks relating to intellectual property; the price of our common stock may be volatile; significant costs as a result of operating as a public company; and any securities class action litigation. These and other important factors discussed under the caption "Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended
|Investor Contact:||Media Contact:|
|Theresa McNeely||Cara Mayfield|
|SVP, Corporate Communications||Director, Corporate Communications|
|& Patient Advocacy
Source: Homology Medicines, Inc.